Visionaries: One Harvard Scientist's Quest To Find A Cure For His Kids10:24

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Many scientists believe that human embryonic stem cells — basic cells that can develop into any tissue or organ in the body — hold the key to curing diseases like cancer, diabetes and Parkinson's disease. In the latest installment of our "Visionaries" series, we take a look at the work of a man who has spent his career harnessing the power of stem cells to heal patients.

Doug Melton has been teaching at Harvard for over three decades and is considered one of the premier stem cell scientists in the world. Melton, whose father was a grocery store manager, grew up on Chicago’s South Side. He was the first in his family to go to college.

At the University of Illinois in the 1970s, he majored in the history and philosophy of science. But there was a problem.

"I did not have it in me to be a good philosopher," Melton said.

As an undergraduate, after reading an article by the very first scientist to clone an animal, Melton knew he had found his calling. "When I read that, I thought that was the coolest experiment I had ever heard of," he recalled.

He was so intrigued he went abroad to Cambridge University to study cloning with that same scientist, biologist John Gurdon. There, Melton became a rising academic star who helped create a new approach to the study of how a cell develops. He calls it "the biography of a cell."

"We were very interested in what genes were turned on and off during the life of that cell that made it what it is," Melton said. "It's the same way when you read the biography of a person: What were the influences that affected that person?"

In 1981, Melton was recruited by Harvard, where he focused on molecular biology and embryology. But all that changed 10 years later when Melton's infant son, Sam, became ill. His wife, Gail O'Keefe, says their son, who was 6 months old at the time, was not thriving.

"I started noticing that he was not making eye contact; something was clearly amiss but I couldn’t really put my finger on it," O'Keefe said. "And then one morning he woke up and he was projectile vomiting.”

"We took him to the hospital and for a while no one could really figure out what was wrong with him and it looked quite dire," Melton recalled. "We now know that he was in extreme ketoacidosis, which is a stage before a person goes into a coma."

The couple watched for hours as doctors at Boston Children's Hospital tried to figure out what was wrong with Sam.

"Sam getting the disease made me rethink my priorities in life. ... It gave me a different sort of appreciation for the things we have in life and the things we can so easily lose."

Doug Melton

"And by the quick and clever action of a nurse who checked his urine, recognized that he was diabetic, which was a surprise to the attending physicians and certainly to us," Melton said.

The diagnosis would change the couple’s lives forever. O'Keefe dropped out of graduate school to care for their diabetic baby.

"I mean, taking care of this infant was 24/7," O'Keefe said. "We had to give three injections a day and every thing had to be highly timed. It was just really, really hard."

O’Keefe felt overwhelmed and asked her husband to share more of the burden.

"And he said, ‘What can I do?’ and I said, ‘You are a scientist. We need to find a cure here.' "

Changing Course

Over the next year, Melton transitioned his work from basic biological research to searching for a cure for diabetes.

"Up until that time, I had never really thought a lot about disease; my work didn't really focus on it," Melton said. "Sam getting the disease made me rethink my priorities in life."

In the late 1990s, scientists made a major discovery: They developed stem cells from human embryos. The hope was that these basic human cells could be coaxed into becoming replacement tissues and organs to treat many difficult diseases. Melton now had a powerful new tool.

"It's actually a very simple idea: We have a stem cell that can make anything and we want to use our knowledge about genes and genetic manipulation to tell that cell what to do," he said.

Making A Case For Stem Cell Research

In Melton's lab on the Harvard campus, scientists are manipulating stem cells to develop treatments for diseases like cancer, ALS and diabetes.

Doug Melton pulls out a tank of axolotl, or Mexican salamanders, in his lab at Harvard. (Jesse Costa/WBUR)

"The cells that we would have stored in these freezers would be human embryonic stem cells that have been differentiated, and they would be at different stages of their development," Melton said. "So you could think of it like being somewhere between kindergarten and an adult; they are kind of teenage cells that haven’t quite been told to become a pancreatic beta cell."

Today, stem cells are at the center of Melton's work. But there was a time when this promising science was threatened.

"I truly was naive. I had no idea that this would get so many people excited and object to something which seemed logical and ethical and, I would even say, morally the right thing to do," Melton said.

In 2001, Melton was summoned for private meetings, first with then-Vice President Dick Cheney and then to the oval office with President George W. Bush to make his case for human embryonic stem cell research.

"I’m not sure I was the best spokesperson for that, but I like to think I made a strong case that this work should go forward, and it should go forward with federal support," he said. "Evidently, I didn’t make the case good enough because the president decided what, in his view, was a sort of compromise."

That compromise allowed federal funding for research on already existing human embryonic stem cell lines, but not for any newly created cell lines. Melton says it was a big step back.

"It didn’t stop human embryonic stem cell research. It did mean that for at least eight years it slowed it to a snail’s pace, and I think that’s just an unfortunate period in our history," he said. "It means that it will be eight years longer before we find cures for diseases."

Two months after the Bush administration’s ban on federal funding for new cell lines, Melton’s 14-year-old daughter, Emma, was also diagnosed with Type 1 diabetes.

"Emma getting the disease just redoubled my efforts," he said. "It just strengthened my commitment to focus my professional life on trying to find a cure for this disease."

Without federal funding for new human embryonic stem cell research, many major scientists moved to countries where the work was fully supported. Throughout this time Harvard stuck by Melton, and with the university's help he raised millions of dollars in private funds to continue his work. In 2004 he co-founded the Harvard Stem Cell Institute.

Slow Progress

These days, Melton and O'Keefe spend a lot of time with undergraduates at the dining hall at Eliot House — the Harvard residence hall where the couple are masters. Melton says they took the job because they are grateful to the university.

"Gail and I are unlikely to ever be able to donate a building to Harvard, and so we thought this would be a way we could give back," Melton said.

Sitting in the library at Eliot House surrounded by his biology books, I asked Melton how much progress has been made in stem cell research. Even though there have been no serious clinical applications yet, he is hopeful that treatments will soon be found.

"We don’t understand all of the causes for these degenerative diseases yet. Stem cells offer us a window into understanding it better than we had before," he said. "It’s hard to know how close we are to the end, but I would say we’re definitely more than halfway there."

At this point, Melton’s own lab has developed insulin-producing cells almost ready to be transplanted into patients with Type 1 diabetes. But Type 1 diabetes is an autoimmune disease, which means any transplanted cells would be attacked by the body. So he’s working on the next frontier.

"We think we have an idea about how to use stem cells to get at the primary cause of autoimmunity," Melton said. "If we can transplant beta cells and block the immune attack, we have de facto cured the disease."

Does he think he will cure the disease that affects both his children?